NCT06041620 is a NA clinical trial of VGB-Ex01 in Thalassemia, Beta, sponsored by Institute of Hematology & Blood Diseases Hospital, China.

Who is sponsoring NCT06041620?

NCT06041620 is sponsored by Institute of Hematology & Blood Diseases Hospital, China.

What drugs are being tested in NCT06041620?

Interventions in NCT06041620: VGB-Ex01.

What condition does NCT06041620 study?

NCT06041620 studies Thalassemia, Beta, Thalassemia Major.

Is NCT06041620 still recruiting?

NCT06041620 is currently recruiting now. Last updated 17 October 2023.

Last reviewed 2023-10-17 · How we verify

NCT06041620

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

Recruiting now NA Last updated 17 October 2023

What this trial tests

NA trial testing VGB-Ex01 in Thalassemia, Beta in 2 participants. Currently enrolling.

Timeline

31 August 2023

Primary endpoint
31 December 2025

30 June 2026

Quick facts

Lead sponsor	Institute of Hematology & Blood Diseases Hospital, China
Phase	NA
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	2
Start date	31 August 2023
Primary completion	31 December 2025
Estimated completion	30 June 2026
Sites	1 location across China

Drugs / interventions tested

VGB-Ex01 — full drug profile →

Conditions studied

Thalassemia, Beta — all drugs for Thalassemia, Beta →
Thalassemia Major — all drugs for Thalassemia Major →

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Who can join

Adults 3 to 35, any sex, with Thalassemia, Beta or Thalassemia Major. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
Laurent M, Geoffroy M, Pavani G, Guiraud S. · · 2024 · cited 45× · PMID 38786024 · DOI 10.3390/cells13100800
Current trends of clinical trials involving CRISPR/Cas systems.
Zhang S, Wang Y, Mao D, Wang Y, et al · · 2023 · cited 35× · PMID 38020120 · DOI 10.3389/fmed.2023.1292452
Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.
Cetin B, Erendor F, Eksi YE, Sanlioglu AD, et al · · 2025 · cited 15× · PMID 40160040 · DOI 10.1017/erm.2025.10
Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies.
Banda A, Impomeni O, Singh A, Baloch AR, et al · · 2024 · cited 8× · PMID 38932365 · DOI 10.3390/vaccines12060636
CRISPR-based therapeutic genome editing for inherited blood disorders.
Levesque S, Bauer DE. · · 2025 · cited 7× · PMID 40659814 · DOI 10.1038/s41573-025-01236-y
Clinical hematopoietic stem cell-based gene therapy.
John T, Czechowicz A. · · 2025 · cited 6× · PMID 40285354 · DOI 10.1016/j.ymthe.2025.04.029
Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy.
Bairqdar A, Karitskaya PE, Stepanov GA. · · 2024 · cited 6× · PMID 39769084 · DOI 10.3390/ijms252413321
Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy.
de Morais CCPL, Correia EM, Bonamino MH, Vasconcelos ZFM. · · 2024 · cited 6× · PMID 39276086 · DOI 10.1089/hum.2024.020

Verify or expand the search:

Other recruiting trials for Thalassemia, Beta

Currently open trials in the same condition.

NCT05736419 — A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an · Phase 2 · recruiting
NCT06314529 — Long-term Follow-up Study of BHC001 for TDT · recruiting
NCT05799118 — Study of the Role of Genetic Modifiers in Hemoglobinopathies · recruiting

Other Institute of Hematology & Blood Diseases Hospital, China trials

Trials by the same sponsor.

NCT06991920 — Immune-targeted Combination With Chemotherapy for Acute Leukemia of Ambiguous Lineage · NA · not yet recruiting
NCT07407010 — BCMA/CD3 Bispecific Antibody as Bridging Therapy Before CAR-T Cell Infusion in RRMM · Phase 1 · not yet recruiting
NCT07407140 — VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML · Phase 3 · not yet recruiting
NCT07454226 — ABL/JAK Inhibitors With Chemotherapy and Venetoclax for Ph-like ALL · NA · not yet recruiting
NCT07490288 — Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML · NA · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT06041620 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Institute of Hematology & Blood Diseases Hospital, China
Last refreshed: 17 October 2023

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06041620.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing